Ryvu Therapeutics discovers and develops small molecule therapies that address high value emerging targets in oncology.

Our pipeline is built from internally-discovered candidates which make use of diverse therapeutic mechanisms, including programs directed at targets in the areas of transcriptional regulation, synthetic lethality, immuno-oncology and immunometabolism.

Ryvu’s two most advanced programs are currently in clinical trials. RVU120 is a selective CDK8 kinase inhibitor in clinical development for the treatment of acute myeloid leukemia and high-risk myelodysplastic syndromes, as well as solid tumors.

SEL24/MEN1703 is a dual PIM/FLT3 kinase inhibitor in clinical development for the treatment of acute myeloid leukemia. Additional programs include synthetic lethality candidates directed at WRN and MTAP deletions as well as immuno-oncology programs focused on STING agonists and HPK1.

  • Clinical
  • Immuno-oncology
    & Immunometabolism
  • Synthetic


RVU120 SEL24/

Clinical Projects

  • RVU120

    CDK8 is a kinase submodule of the mediator complex, involved in both transcriptional activation and repression. CDK8- mediator complex integrates basal transcriptional machinery with the activity of oncogenic transcriptional and epigenetic factors. Targeting CDK8 and...

  • SEL24/MEN1703

    SEL24/MEN1703 is a selective, dual inhibitor of PIM and FLT3 kinases, two enzymes that are strongly implicated in malignant transformation of hematopoietic cells. The compound is a novel small molecule discovered by Ryvu and currently...

Immuno-oncology & Immunometabolism

Ryvu Therapeutics has deep expertise in the discovery of small molecule candidates with optimized properties to address specific limitations of existing therapeutic approaches.

To identify these candidates, Ryvu uses platforms based on the most current understanding of how the immune system interacts with cancer cells and how cancer cells function biologically. These immuno-oncology and immunometabolism approaches have a successful history in biopharmaceutical development. Ryvu has accumulated scientific and technical infrastructure that allow high-throughput generation of candidates using these platforms.


    STING protein is one of the major players in innate immune activation being a potent trigger of type I interferon (IFN) and pro-inflammatory cytokines. STING agonists facilitate cancer antigen recognition specific to individual patient and...


    The high therapeutic potential of HPK1 inhibitors (hematopoietic progenitor kinase 1, MAP4K1) lies in their potential to simultaneously improve T cell function, tumor antigen presentation and combat the immunosuppressive tumor microenvironment.  HPK1 is a negative...

Synthetic Lethality

One of the challenges in the development of new oncology therapeutics is the limited number of protein targets known to be addressable with small molecules. Synthetic lethality allows the identification of novel protein targets and mechanisms of cancer cell sensitivity that can be used therapeutically.

Compounds active on specific synthetic lethal targets selectively kill tumor cells of a precisely defined model system, characterized by a specific genetic, epigenetic or metabolic characteristic. This is the approach actively investigated at Ryvu that allows the generation of compounds with highly specific and well-characterized activity.


    Deletion of 9p21 locus is one of the most frequent genetic alterations associated with human cancers. Apart from tumor suppressor p16, locus 9p21 contains the gene coding methylthioadenosine phosphorylase (MTAP), the only enzyme capable of...

Novel Targets

Beyond publicly disclosed programs, Ryvu is working on candidates that address novel targets in the areas of synthetic lethality, immuno-oncology and immunometabolism to generate new therapeutics with first-in-class or best-in-class potential.

For the candidates, Ryvu is utilizing a proprietary drug discovery approach combining internally developed expertise and new sophisticated tools and technologies to identify small molecule compounds with favorable profiles and activity.

Discovery Collaborations

In April 2020, Ryvu Therapeutics and Galapagos have entered into a collaboration focused on the discovery and development of novel small molecule drugs in inflammation.
The collaboration is based on a novel drug target identified by Ryvu, which will contribute its technology platform and related intellectual property. Ryvu and Galapagos will both provide resources to support the collaboration and make use of their expertise in high-throughput screening, biology, medicinal chemistry, and toxicology.
During the joint research collaboration, Ryvu is responsible for early drug discovery and Galapagos will be responsible for all further development of the program.

In 2013, Ryvu and Merck KGaA initiated a joint research strategy involving intensive work on multiple targets and several chemical series. The collaboration has successfully completed two milestones and has been expanded over time with additional targets.
The aim of the partnership with Merck is the development of new anticancer therapeutics acting on diverse biological targets associated with aberrant metabolic pathways in cancer cells. Dependence on specific metabolic pathways is a feature of various types of cancer, therefore, targeting this vulnerability has many potential applications in a broad range of patient populations.
The collaboration has resulted in scientific achievements, the joint filing of patent applications and data presentations at international scientific conferences.


Society for Immunotherapy of Cancer’s (SITC) 36th Anniversary Annual Meeting, November 11-14, 2021

  • "Novel, orally administered HPK1 inhibitors demonstrate anti-tumor efficacy and enhanced immune response"

    PDF Download
  • "Characterization of RVU-27065 a novel small-molecule STING agonist suitable for systemic administration"

    PDF Download

Discovery of 5-{2-[5-Chloro-2-(5-ethoxyquinoline-8-sulfonamido)phenyl]ethynyl}-4-methoxypyridine-2-carboxylic Acid, a Highly Selective in Vivo Useable Chemical Probe to Dissect MCT4 Biology

  • Heinrich T, Sala-Hojman A, Ferretti R, Petersson C, Minguzzi S, Gondela A, Ramaswamy S, Bartosik A et al. Discovery of 5-{2-[5-Chloro-2-(5-ethoxyquinoline-8-sulfonamido)phenyl]ethynyl}-4-methoxypyridine-2-carboxylic Acid, a Highly Selective in Vivo Useable Chemical Probe to Dissect MCT4 Biology. Journal of Medicinal Chemistry, August 2021

    PDF Download

Virtual 26th Annual European Hematology Association (EHA) Congress, June 9-17, 2021

  • "RVU120/SEL120 CDK8/19 inhibitor - a drug candidate for the treatment of MDS can induce erythroid differentiation in transformed CD34+ hematopoietic progenitor cells"

    PDF Download
  • "CLI120-001 Phase 1b Study of SEL120/RVU120 in patients with AML or High Risk MDS: Preliminary clinical and PK results from initial dose escalation cohorts"

    PDF Download
  • “Results from DIAMOND-01 (CLI24-001) TRIAL: First in Human Study of SEL24/MEN1703, a Dual PIM/FLT3 Kinase Inhibitor, in Patients with Acute Myeloid Leukemia”

    PDF Download

AACR Virtual Annual Meeting 2021, April 10-15 and May 17-21, 2021

  • "RVU120 (SEL120), a CDK8/CDK19 inhibitor, possesses strong multilineage differentiation potential in leukemic cells"

    PDF Download
  • "Development and characterization of small molecule HPK1 inhibitors"

    PDF Download
  • "New generation of STING agonists: development and characterization of a novel series of systemic immunomodulators with improved potency"

    PDF Download